Gene specific therapies

• Verfasst von: Brenner, David [VerfasserIn]
• Verfasst von: Ludolph, Albert C. [VerfasserIn]
• Verfasst von: Weishaupt, Jochen H. [VerfasserIn]
• Titel: Gene specific therapies
• Titelzusatz: the next therapeutic milestone in neurology
• Verf.angabe: David Brenner, Albert C. Ludolph and Jochen H. Weishaupt
• E-Jahr: 2020
• Jahr: 2020 Sept. 8
• Umfang: 13 S.
• Fussnoten: Gesehen am 07.04.2021
• Titel Quelle: Enthalten in: Neurological research and practice
• Ort Quelle: [London] : BioMed Central, 2019
• Jahr Quelle: 2020
• Band/Heft Quelle: 2(2020), Artikel-ID 25, Seite 1-13
• ISSN Quelle: 2524-3489
• DOI: doi:10.1186/s42466-020-00075-z
• Datenträger: Online-Ressource
• Sprache: eng
• K10plus-PPN: 1753282519

Abstract

Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges.